Peg-ada ert (DrugBank: -)
1 diseaseID | Disease name (Link within this page) | Number of trials |
---|---|---|
65 | Primary immunodeficiency | 2 |
No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
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1 | NCT02999984 (ClinicalTrials.gov) | December 16, 2016 | 19/12/2016 | Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID | Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency | Severe Combined Immunodeficiency Due to ADA Deficiency | Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: busulfan;Drug: PEG-ADA ERT | Orchard Therapeutics | California Institute for Regenerative Medicine (CIRM);University of California, Los Angeles | Completed | N/A | 17 Years | All | 10 | Phase 1;Phase 2 | United States |
2 | NCT01852071 (ClinicalTrials.gov) | August 2, 2013 | 7/5/2013 | Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With EFS Lentiviral Vector Encoding for the Human ADA Gene | Autologous Transplantation of Bone Marrow CD34+ Stem/Progenitor Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID) | ADA-SCID | Genetic: Infusion of autologous EFS-ADA LV CD34+ (OTL-101);Drug: busulfan;Drug: PEG-ADA ERT | Orchard Therapeutics | National Institute of Allergy and Infectious Diseases (NIAID);National Human Genome Research Institute (NHGRI);National Heart, Lung, and Blood Institute (NHLBI);University of California, Los Angeles | Completed | 1 Month | 17 Years | All | 20 | Phase 1;Phase 2 | United States |